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WO2025097067A3 - Engineered cell preparations for treatment of hypophosphatasia - Google Patents

Engineered cell preparations for treatment of hypophosphatasia Download PDF

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Publication number
WO2025097067A3
WO2025097067A3 PCT/US2024/054285 US2024054285W WO2025097067A3 WO 2025097067 A3 WO2025097067 A3 WO 2025097067A3 US 2024054285 W US2024054285 W US 2024054285W WO 2025097067 A3 WO2025097067 A3 WO 2025097067A3
Authority
WO
WIPO (PCT)
Prior art keywords
hypophosphatasia
treatment
engineered cell
cell preparations
alp
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
PCT/US2024/054285
Other languages
French (fr)
Other versions
WO2025097067A9 (en
WO2025097067A2 (en
Inventor
Hanlan Liu
Sarah Grace LEACH
JR. Wayne BAINTER
Madison Ashley CLEMENTS
Richard A. Morgan
Timothy James MULLEN
Anja Fides Hohmann
Shamael Rabia Dastagir
Sharvari GUJJA
Sean Patrick KEEGAN
Andre STANLIE
Toshiro Kendrick Ohsumi
Monika MUSIAL-SIWEK
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Be Biopharma Inc
Original Assignee
Be Biopharma Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Be Biopharma Inc filed Critical Be Biopharma Inc
Publication of WO2025097067A2 publication Critical patent/WO2025097067A2/en
Publication of WO2025097067A3 publication Critical patent/WO2025097067A3/en
Publication of WO2025097067A9 publication Critical patent/WO2025097067A9/en
Pending legal-status Critical Current
Anticipated expiration legal-status Critical

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/10—Cellular immunotherapy characterised by the cell type used
    • A61K40/13—B-cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/30—Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0635—B lymphocytes
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/30—Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/20—Cytokines; Chemokines
    • C12N2501/23—Interleukins [IL]
    • C12N2501/2302—Interleukin-2 (IL-2)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/20—Cytokines; Chemokines
    • C12N2501/23—Interleukins [IL]
    • C12N2501/231—Interleukin-10 (IL-10)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/20—Cytokines; Chemokines
    • C12N2501/23—Interleukins [IL]
    • C12N2501/2315—Interleukin-15 (IL-15)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/20—Cytokines; Chemokines
    • C12N2501/23—Interleukins [IL]
    • C12N2501/2321—Interleukin-21 (IL-21)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/50—Cell markers; Cell surface determinants
    • C12N2501/52—CD40, CD40-ligand (CD154)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00—Genetically modified cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/03—Phosphoric monoester hydrolases (3.1.3)
    • C12Y301/03001—Alkaline phosphatase (3.1.3.1)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Immunology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Hematology (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Molecular Biology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)

Abstract

The present disclosure provides methods and compositions for culturing, engineering, and administering engineered B lineage cell populations that produce an ALP or a fusion protein thereof, such as ALP-Fc fusion protein comprising alkaline phosphatase and human immunoglobulin fragment crystallizable region.
PCT/US2024/054285 2023-11-02 2024-11-01 Engineered cell preparations for treatment of hypophosphatasia Pending WO2025097067A2 (en)

Applications Claiming Priority (10)

Application Number Priority Date Filing Date Title
US202363595741P 2023-11-02 2023-11-02
US63/595,741 2023-11-02
US202463623768P 2024-01-22 2024-01-22
US63/623,768 2024-01-22
US202463643222P 2024-05-06 2024-05-06
US63/643,222 2024-05-06
US202463660968P 2024-06-17 2024-06-17
US63/660,968 2024-06-17
US202463698944P 2024-09-25 2024-09-25
US63/698,944 2024-09-25

Publications (3)

Publication Number Publication Date
WO2025097067A2 WO2025097067A2 (en) 2025-05-08
WO2025097067A3 true WO2025097067A3 (en) 2025-07-10
WO2025097067A9 WO2025097067A9 (en) 2025-08-07

Family

ID=95583311

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2024/054285 Pending WO2025097067A2 (en) 2023-11-02 2024-11-01 Engineered cell preparations for treatment of hypophosphatasia

Country Status (1)

Country Link
WO (1) WO2025097067A2 (en)

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018170150A2 (en) * 2017-03-16 2018-09-20 Seattle Children's Hospital (dba Seattle Children's Research Institute) Engraftable cell-based immunotherapy for long-term delivery of therapeutic proteins
US10449236B2 (en) * 2014-12-05 2019-10-22 Alexion Pharmaceuticals, Inc. Treating seizure with recombinant alkaline phosphatase
WO2022051555A2 (en) * 2020-09-03 2022-03-10 Rampart Bioscience, Inc. Soluble alkaline phosphatase constructs and expression vectors including a polynucleotide encoding for soluble alkaline phosphatase constructs
WO2024220446A2 (en) * 2023-04-17 2024-10-24 Be Biopharma, Inc. Engineered cell preparations for treatment of hemophilia

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10449236B2 (en) * 2014-12-05 2019-10-22 Alexion Pharmaceuticals, Inc. Treating seizure with recombinant alkaline phosphatase
WO2018170150A2 (en) * 2017-03-16 2018-09-20 Seattle Children's Hospital (dba Seattle Children's Research Institute) Engraftable cell-based immunotherapy for long-term delivery of therapeutic proteins
WO2022051555A2 (en) * 2020-09-03 2022-03-10 Rampart Bioscience, Inc. Soluble alkaline phosphatase constructs and expression vectors including a polynucleotide encoding for soluble alkaline phosphatase constructs
WO2024220446A2 (en) * 2023-04-17 2024-10-24 Be Biopharma, Inc. Engineered cell preparations for treatment of hemophilia

Also Published As

Publication number Publication date
WO2025097067A9 (en) 2025-08-07
WO2025097067A2 (en) 2025-05-08

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